Experimental drug denied to critically-ill seven-year-old

Josh Hardy is in critical condition in an intensive care unit in Memphis. The 7-year-old is in heart and kidney failure. He vomits blood several times an hour.

Josh’s was diagnosed with a rare form of kidney cancer at 9 months old. Over the years, cancer turned up in his thymus, lung, and bone marrow. A bone marrow transplant left him without much of an immune system, and in February doctors diagnosed him with an adenovirus that spread through his body.

There is a new drug that could possibly help Josh but it is not yet released to the public. Under the “compassionate use” program it is possible for a patient ask a drug company for an experimental drug, one that they’re still studying and has not yet been approved by the Food and Drug Administration (FDA), but pharmaceutical companies often say no, as they did to Josh Hardy.

Chimerix makes the drug and is working to get it on the market by the end of 2016. They say if they spend time and money on compassionate use cases, it would greatly hinder their effort to get the drug, brincidofovir, on the market and available to everyone.
Since insurance usually does not pay for experimental drugs the company would have to cover the $50,000 per compassionate-use per patient, says company president Kenneth Moch said. And perhaps even more important than the money, it would divert manpower in this 50-person company, since they’d have to handle the requests and then get the patient’s records and follow up with them, as required by the FDA.

And Josh might not be the only one needing brincidofovir. “If this were just one patient wanting this drug, then this would be a very different question,” Moch said. “But it’s yes to all or no to all.”

Art Caplan, a bioethicist at NYU Langone Medical Center says it’s not just the $50,000 per patient that might make investors squeamish. But compassionate cases can make a drug look bad. By definition, compassionate use patients are extremely sick, and might not do well with the drug. Companies have to report that poor outcome to the FDA in its application to market the drug.